WAITING FOR EVIDENCE OR EARLIER ACCESS? PREFERENCES FOR ONE-TIME THERAPIES AMONG CANCER PATIENTS AND THE GENERAL PUBLIC
Author(s)
Sojeong Bae, PharmD1, Gyeongseon Shin, PharmD, PhD2, Seungjin Bae, ScD1.
1College of Pharmacy, Ewha Womans University, Seoul, Korea, Republic of, 2College of Pharmacy, Wonkwang University, Iksan, Korea, Republic of.
1College of Pharmacy, Ewha Womans University, Seoul, Korea, Republic of, 2College of Pharmacy, Wonkwang University, Iksan, Korea, Republic of.
OBJECTIVES: High-cost one-time therapies, such as CAR-T and gene therapies, offer potentially lifelong benefits with a single administration, yet limited follow-up leaves long-term effectiveness uncertain. Combined with substantial financial burdens, immature evidence creates challenges for reimbursement and coverage decisions. This study quantified willingness-to-wait (WTW) for evidence maturity and attribute importance among Korean cancer patients and general population.
METHODS: A web-based discrete choice experiment (DCE) and case 2 best-worst scaling (BWS) were conducted. Four attributes were included: long-term evidence maturity, serious adverse event rate, waiting time, and out-of-pocket costs. Three overall survival (OS) scenarios (36-, 18-, 6-month) and three decision contexts (self, child, elderly) were examined. DCE data were analyzed using mixed logit models in WTW space, while BWS was used to assess attribute importance.
RESULTS: Among 2,668 respondents, 2,432 passed the dominance check and were included (1,969 general population; 463 cancer patients). WTW for evidence maturity declined as the OS scenario shortened. Under the longest scenario (36-month OS), the general population WTW for 12→60-month evidence reached 24.03 months, with comparable estimates in cancer patients (26.55 months). This attenuated under the 18-month OS scenario (5.53 and 6.07 months, respectively). Under the 6-month OS scenario, both groups showed negative WTW for the smallest evidence increment (12→18 months: −0.83 months -2.61 months, respectively), indicating a preference for immediate access over additional evidence. WTW to avoid the highest adverse event rate exceeded life expectancy across all OS scenarios, indicating strong aversion to high-toxicity options. BWS showed a consistent attribute ranking across groups (cost > adverse event > waiting time > evidence maturity).
CONCLUSIONS: The value placed on evidence maturity decreased markedly as prognosis worsened, indicating context-dependent preferences for uncertainty reduction. Delaying access to obtain additional long-term evidence may not align with preferences under poor-prognosis conditions. These findings support flexible approaches to evidence generation and access for one-time therapies.
METHODS: A web-based discrete choice experiment (DCE) and case 2 best-worst scaling (BWS) were conducted. Four attributes were included: long-term evidence maturity, serious adverse event rate, waiting time, and out-of-pocket costs. Three overall survival (OS) scenarios (36-, 18-, 6-month) and three decision contexts (self, child, elderly) were examined. DCE data were analyzed using mixed logit models in WTW space, while BWS was used to assess attribute importance.
RESULTS: Among 2,668 respondents, 2,432 passed the dominance check and were included (1,969 general population; 463 cancer patients). WTW for evidence maturity declined as the OS scenario shortened. Under the longest scenario (36-month OS), the general population WTW for 12→60-month evidence reached 24.03 months, with comparable estimates in cancer patients (26.55 months). This attenuated under the 18-month OS scenario (5.53 and 6.07 months, respectively). Under the 6-month OS scenario, both groups showed negative WTW for the smallest evidence increment (12→18 months: −0.83 months -2.61 months, respectively), indicating a preference for immediate access over additional evidence. WTW to avoid the highest adverse event rate exceeded life expectancy across all OS scenarios, indicating strong aversion to high-toxicity options. BWS showed a consistent attribute ranking across groups (cost > adverse event > waiting time > evidence maturity).
CONCLUSIONS: The value placed on evidence maturity decreased markedly as prognosis worsened, indicating context-dependent preferences for uncertainty reduction. Delaying access to obtain additional long-term evidence may not align with preferences under poor-prognosis conditions. These findings support flexible approaches to evidence generation and access for one-time therapies.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
PT18
Topic
Health Policy & Regulatory, Patient-Centered Research
Disease
Genetic, Regenerative & Curative Therapies, Oncology